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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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16 December 2017 |
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Main ID: |
NCT01451879 |
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Date of registration:
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15/09/2011 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
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Scientific title:
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Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy |
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Date of first enrolment:
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October 2008 |
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Target sample size:
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11 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01451879 |
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Study type:
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Observational |
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Study design:
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Phase:
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N/A
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Countries of recruitment
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United States
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Contacts
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Name:
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Barry J Byrne, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Florida |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- patients between the ages of 0 months and 65 years
- diagnosed with early-onset Pompe Disease, confirmed by mutational analysis and/or GAA
enzyme assay
- eligible regardless of whether they have begun enzyme replacement therapy prior to
enrollment
- all subjects will receive ERT as standard of care during the course of the study,
although they may not have begun ERT treatment at the time of enrollment
- subjects may receive an immunomodulatory regimen as part of their standard of care;
this may include rituximab, sirolimus, methotrexate, Gamunex, Hizentra, Zavesca or
other immunomodulatory agents, alone or in combination, at the discretion of their
caregiver(s)
Exclusion Criteria:
- subject is unable to meet the study requirements
- subjects medical condition contraindicates participation or Study Investigators feel
that participation is otherwise not in the subject's best interest
- subject does not receive ERT treatment
Age minimum:
N/A
Age maximum:
65 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pompe Disease
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Intervention(s)
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Drug: Miglustat
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Drug: Rituximab
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Primary Outcome(s)
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Anti-rh GAA antibody titers
[Time Frame: 52 weeks]
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Secondary Outcome(s)
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B-lymphocyte antigen (CD20) level
[Time Frame: 52 weeks]
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Secondary ID(s)
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IMN 439-2008
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IRB201602404
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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